Healthcare Industry News:  GCS-100 

Biopharmaceuticals Neurology FDA

 News Release - July 17, 2013

La Jolla Pharmaceutical Company Receives Orphan Designation From FDA

SAN DIEGO, CA--(Healthcare Sales & Marketing Network) - La Jolla Pharmaceutical Company (OTCBB: LJPC) ("La Jolla" and "Company"), a leader in the development of therapeutics targeting life threating diseases, announced today that the FDA Office of Orphan Products Development has granted an "Orphan Drug" designation to La Jolla's drug candidate LJPC-0712 for treatment of Niemann-Pick type C disease.

"Having received a second orphan designation from FDA, our team continues to build La Jolla into a leader in the development of treatments for life threating diseases," said George Tidmarsh, MD, PhD, President and Chief Executive Officer of La Jolla. "We remain dedicated to developing novel treatments to help those suffering with unmet debilitating and fatal diseases."

Niemann-Pick type C disease (NP-C) is a rare inherited disease caused by mutations in either the NPC1 (95% of cases) or of the NPC2 gene. These mutations lead to impaired intracellular lipid trafficking with subsequent accumulation of cholesterol and glycosphingolipids in the brain and other tissues. Patients come to medical attention with a range of symptoms, neurologic signs, age of onset and rate of progression. Characteristic neurological manifestations include abnormal eye movements, unbalanced gait, abnormal speech, or difficulty swallowing. Epileptic seizures are also common. Typically, onset occurs in childhood, however adults are increasingly being diagnosed with late onset neurologic symptoms or psychiatric manifestations. The age of onset of neurologic symptoms is used to categorize patients and can be useful in evaluation and treatment planning. In general, patients diagnosed earlier in age progress more rapidly and have a shorter survival than those diagnosed later in life. Patients with NP-C have an estimated median survival of less than 20 years.

LJPC-0712 is commonly known as allopregnanolone. Allopregnanolone is a neurosteroid present in the blood and brain. It is a metabolite of progesterone and potent modulator of gamma-aminobutyric acid (GABAA) receptors. It increases the activity of endogenous GABA by allosteric modification of the GABAA receptor. Allopregnanolone has pharmacological properties similar to other positive modulators of GABAA receptors, including anti-anxiety and anti-seizure activity.

Allopregnanolone is synthesized from cholesterol and due to disrupted cholesterol biosynthesis levels are altered in NP-C. Because allopregnanolone and related steroid hormones promote neuron health and development, reduced levels of allopregnanolone in NP-C likely contribute to the neurologic deterioration associated with NP-C. End-product replacement therapy targets the final products of a metabolic pathway that are deficient in a disease state. Therefore, replacement therapy with allopregnanolone represents a potential treatment for NP-C. Treatment of animals genetically modified to replicate NP-C with allopregnanolone supports this theory with treated animals showing improvement in many disease characteristics.

About La Jolla Pharmaceutical Company

La Jolla Pharmaceutical Company is a biopharmaceutical company dedicated to the development of medical treatments that significantly improve outcomes in patients with life-threatening diseases. GCS-100, the Company's lead product candidate, is a first-in-class inhibitor of galectin-3, a novel molecular target implicated in chronic organ failure and cancer, currently being tested in a Phase 2 clinical trial for the treatment of chronic kidney disease. For more information on the Company please visit

Forward-Looking Statement Safe Harbor

This document contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements relate to future events or our future results of operations. These statements are only predictions and involve known and unknown risks, uncertainties and other factors, which may cause actual results to be materially different from these forward-looking statements. The Company cautions readers not to place undue reliance on any such forward-looking statements, which speak only as of the date they were made. Certain of these risks, uncertainties, and other factors are described in greater detail in the Company's filings from time to time with the U.S. Securities and Exchange Commission ("SEC"), all of which are available free of charge on the SEC's web site These risks include, but are not limited to, risks relating to the development of GCS-100 and LJPC-501, the success and timing of future preclinical and clinical studies of this compound, and potential indications for which GCS-100 and LJPC-501 may be developed. Subsequent written and oral forward-looking statements attributable to the Company or to persons acting on its behalf are expressly qualified in their entirety by the cautionary statements set forth in the Company's reports filed with the SEC. The Company expressly disclaims any intent to update any forward-looking statements.

Source: La Jolla Pharmaceutical

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